Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

NCT01903460 | Completed Phase 2 Results DMC

• 3 other identifiers: LUM001-3022012-005346-38SHP625-302
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

Brief Summary

The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

Conditions

Alagille Syndrome

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level

  Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.

  Baseline to 13 weeks or end of treatment

Secondary Outcomes (2)

• Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes

  Baseline to 13 weeks or end of treatment

• Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores

  Baseline to 13 weeks or end of treatment

Study Arms (2)

LUM001

EXPERIMENTAL

LUM001 administered orally once each day

Drug: LUM001

Placebo

PLACEBO COMPARATOR

Placebo administered orally once each day

Drug: Placebo

Interventions

LUM001 DRUG

LUM001

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 12 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of Alagille Syndrome
• Evidence of cholestasis
• Moderate to severe pruritus
• Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures

You may not qualify if:

• Surgical disruption of the enterohepatic circulation
• Liver transplant
• History or presence of other concomitant liver disease
• Females who are pregnant or lactating
• Known HIV infection

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (3)

Birmingham Children's Hospital

Birmingham, West Midlands, B4 6NH, United Kingdom

Location

Leeds Teaching Hospitals

Leeds, West Yorkshire, LS9 7TF, United Kingdom

Location

Kings College Hospital

London, SE5 9RS, United Kingdom

Location

MeSH Terms

Conditions

Alagille Syndrome

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Results Point of Contact

• Title: Study Physician
• Organization: Mirum

medinfo@mirumpharma.com

+1 650-667-4085

Study Officials

• Study Director

  Mirum

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2013
• First Posted: July 19, 2013
• Study Start: August 1, 2013
• Primary Completion: February 1, 2015
• Study Completion: March 1, 2015
• Last Updated: March 28, 2019
• Results First Posted: December 7, 2015

Record last verified: 2019-03

Locations

GB (3)